Clinical efficacy and safety of single cycle rituximab as induction therapy for aggressive neuromyelitis optica spectrum disorder in a resource limited center: a preliminary study

摘要

Aim: To analyse the efficacy of single dose rituximab (RTX) as induction therapy followed by conventional oral steroid-sparing agents (azathioprine, mycophenolate mofetil or methotrexate) in a cohort of patients with aggressive neuromyelitis optica spectrum disorder (NMOSD) without CD19, 20 and 27 biomarker testing.Methods: A retrospective analysis of clinical outcomes in eight patients with aggressive NMOSD treated with one course of RTX induction therapy in the Neurology Department at Kuala Lumpur Hospital from 2005 to 2018 was performed. The effectiveness of the treatment was determined by the number of relapses, expanded disability status scale, annualized relapsed rates, and modified Rankin Scale both before and after treatment. B cell enumeration testing was done instead of CD19, 20 and 27 biomarker testing. Results: There was a reduction in the mean annualized relapse rate from 4.7 to 0.5 attacks per year after treatment (P = 0.011). Mean expanded disability status scale and modified Rankin Scale values improved from 5.4 to 3.6 (P = 0.018) and 3.6 to 2.6 (P = 0.023), respectively. No patient developed any adverse effect. Conclusion: Single-course RTX induction therapy regime may be an alternative therapeutic option in resource limited hospitals to suppress NMOSD disease activity in the short term as pulse induction therapy whilst awaiting the effectiveness of conventional steroid-sparing agents. Further prospectively designed studies are required to prove efficacy.